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视网膜类器官在疾病建模和药物发现中的应用:机遇与挑战。

Retinal organoids in disease modeling and drug discovery: Opportunities and challenges.

机构信息

GROW Research Laboratory, Narayana Nethralaya Foundation, Bangalore, Karnataka, India.

GROW Research Laboratory, Narayana Nethralaya Foundation, Bangalore, Karnataka, India.

出版信息

Surv Ophthalmol. 2024 Mar-Apr;69(2):179-189. doi: 10.1016/j.survophthal.2023.09.003. Epub 2023 Sep 29.

DOI:10.1016/j.survophthal.2023.09.003
PMID:37778668
Abstract

Diseases leading to retinal cell loss can cause severe visual impairment and blindness. The lack of effective therapies to address retinal cell loss and the absence of intrinsic regeneration in the human retina leads to an irreversible pathological condition. Progress in recent years in the generation of human three-dimensional retinal organoids from pluripotent stem cells makes it possible to recreate the cytoarchitecture and associated cell-cell interactions of the human retina in remarkable detail. These human three-dimensional retinal organoid systems made of distinct retinal cell types and possessing contextual physiological responses allow the study of human retina development and retinal disease pathology in a way animal model and two-dimensional cell cultures were unable to achieve. We describe the derivation of retinal organoids from human pluripotent stem cells and their application for modeling retinal disease pathologies, while outlining the opportunities and challenges for its application in academia and industry.

摘要

导致视网膜细胞损失的疾病可导致严重的视力损害和失明。目前缺乏有效的治疗方法来解决视网膜细胞损失的问题,而且人类视网膜中缺乏内在的再生能力,导致了不可逆转的病理状况。近年来,多能干细胞生成人类三维视网膜类器官的进展使得以惊人的细节重现人类视网膜的细胞结构和相关的细胞间相互作用成为可能。这些由不同视网膜细胞类型组成的人类三维视网膜类器官系统具有上下文生理反应,使得可以以动物模型和二维细胞培养无法实现的方式研究人类视网膜的发育和视网膜疾病的病理。我们描述了从人类多能干细胞中衍生出的视网膜类器官及其在模拟视网膜疾病病理中的应用,同时概述了其在学术界和工业界的应用的机会和挑战。

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